elivaldogene autotemcel

Elivaldogene autotemcel is a gene therapy used to treat early, active cerebral adrenoleukodystrophy (CALD) in males aged 4 to 17 years. CALD is a rare X-linked genetic disorder caused by mutations in the ABCD1 gene, leading to accumulation of very long-chain fatty acids (VLCFAs) in the brain and nervous system. This buildup results in progressive demyelination and neurodegeneration. Elivaldogene autotemcel works by collecting autologous hematopoietic stem cells from the patient, transducing them ex vivo with a lentiviral vector carrying a functional copy of the ABCD1 gene (Lenti-D), and reinfusing these modified cells back into the patient after myeloablative conditioning. The engrafted cells express functional ALDP protein, restoring peroxisomal beta-oxidation capacity and enabling breakdown of VLCFAs, thereby slowing or stabilizing disease progression.

Brand names

Skysona

Other names

Lenti-DLenti-D LVV